EUROPEAN CYSTIC FIBROSIS SOCIETY – CLINICAL TRIALS NETWORK ECFS-CTN reporting to patients 2014 European Cystic Fibrosis Society – Clinical Trials Network [email protected] ECFS-CTN reporting to patients 2014 Content GENERAL REPORTING: ............................................................................................................................................ 3 1. WHY ARE CLINICAL TRIALS IMPORTANT? ..................................................................................................................... 3 2. WHAT IS THE ECFS-CTN AND WHICH SITES ARE INVOLVED? ........................................................................................... 3 3. WHAT DOES THE ECFS-CTN DO? ............................................................................................................................. 4 4. WHAT IS THE DIRECT AND POTENTIAL BENEFIT TO PATIENTS? .......................................................................................... 8 5. ECFS-CTN FINANCIAL SUPPORT ............................................................................................................................... 8 6. WHY DOES THE ECFS-CTN NEED YOUR SUPPORT AND WHAT CAN YOU DO TO SUPPORT ECFS-CTN? .................................... 9 2014 ANNUAL REPORTING ................................................................................................................................... 11 1. PARTICIPATING SITES, GOVERNANCE, COMMITTEES ..................................................................................................... 11 1.1. ECFS-CTN sites 2014: ............................................................................................................................. 11 1.2. Coordinating centre: ............................................................................................................................... 12 1.3. Governance: ........................................................................................................................................... 13 1.4. Committees: ........................................................................................................................................... 15 2. ACTIVITIES OF ECFS-CTN IN 2014 ......................................................................................................................... 15 2.1. Studies (clinical trials) ............................................................................................................................. 15 2.2. Training .................................................................................................................................................. 21 2.3. Standardisation ...................................................................................................................................... 21 2.4. Patient involvement ............................................................................................................................... 24 2.5. Quality program and evaluation of sites ................................................................................................ 24 2.6. Investigator Initiated Trial ...................................................................................................................... 25 3. FINANCIAL REPORT 2014 ...................................................................................................................................... 26 4. SHORT SUMMARY OF FUTURE CTN PROJECTS: ........................................................................................................... 27 1 European Cystic Fibrosis Society – Clinical Trials Network [email protected] ECFS-CTN reporting to patients 2014 Abbreviations Abbreviations used in this Report: CF Cystic Fibrosis CFF-TDN Cystic Fibrosis Foundation Therapeutics Development Network (CF Clinical Trial Network established in USA) CFTR Cystic Fibrosis Trans-membrane Regulator protein CTN Clinical Trial Network CRO Clinical Research Organisation (often these companies act to administer the set-up and running of a clinical trial on behalf of pharmaceutical companies) ECFS European Cystic Fibrosis Society GCP Good Clinical Practice (this is the ethical foundation to protect the rights and welfare of all volunteers in clinical trials) ICM Intestinal Current Measurement (a measurement of chloride transport across the skin lining the inside of the bowel – a good measurement of how well CFTR is working and used as a measure of effectiveness of some new treatments in clinical trials) LCI Lung Clearance Index (this is a sensitive type of lung function measurement that measures how long it takes for a gas (usually nitrogen) to be washed out of the lungs during normal breathing and used as a measure of effectiveness of some new treatments in clinical trials) NPD Nasal Potential Difference (a measurement of chloride transport across the skin lining the inside of the nose – a good measurement of how well CFTR is working and used as a measure of effectiveness of some new treatments in clinical trials) SOP Standard Operating Procedure TMS Trial Management System (a web-based reporting system that allows site teams to demonstrate how each clinical trial is running at their site) 2 European Cystic Fibrosis Society – Clinical Trials Network [email protected] ECFS-CTN reporting to patients 2014 ECFS-CTN Reporting to Patients General reporting: 1. Why are clinical trials important? A clinical trial is a research study in human volunteers to answer specific health questions, for example to test if new potential treatments are safe and effective. “Effective” means the drug works better or as well as the currently used treatment and helps the patient feel better. Carefully conducted clinical trials are the safest, fastest and only authorized way to develop new and effective treatments. In 2012, a milestone was reached in the fight against cystic fibrosis (CF). For the first time ever, a drug aimed at treating the cause of CF, the abnormal CFTR protein, was approved for marketing in Europe and the US. This novel ‘disease-modifying drug’, Ivacaftor, is now available to patients because its safety and efficacy have been proven in clinical trials. While this drug only works in a small minority of CF patients, other drugs with new mechanisms of action potentially working in the majority of patients are currently being investigated in clinical trials. Furthermore, and thanks to the tremendous mobilization of the CF community in the past years, many drugs have shown potential for treating infection, inflammation and digestive troubles occurring in CF. Increasingly clinical trials are assessing these new treatments in younger children with CF too, to ensure safety, as in many cases starting treatments earlier in life may further improve life expectancy and quality of life. This significant progress in life expectancy in CF could not have been established without clinical trials. 2. What is the ECFS-CTN and which sites are involved? With aim to help developing new drugs for CF most safely and most efficiently, the European Cystic Fibrosis Society (ECFS), a scientific organisation aiming at advancing CF care and research throughout Europe and beyond, has taken the initiative to form a “European Clinical Trials Network”, the “ECFS-CTN”, a development that has been strongly supported by the European CF patient organisations. The aim of this network is to implement high quality clinical research in order to facilitate the progress of drugs from clinical trials to the patient. For a rare disease such as CF, it is important that the whole CF community is mobilized and that countries work together as much as possible. Also, there is a need for a 3 European Cystic Fibrosis Society – Clinical Trials Network [email protected] ECFS-CTN reporting to patients 2014 close cooperation between patients, patient organisations, pharmaceutical industry and academic research institutions. In 2014, this network includes CF specialists from 30 large CF centres in 11 countries (Belgium, Czech Republic, Denmark, France, Germany, Italy, Portugal, Spain, Sweden, The Netherlands and the UK). The complete list of centres is available on the ECFS-CTN website: http://www.ecfs.eu/ctn/list-ctn-centres and further in this document. With these 30 centres, the network gathers 14,000 CF children and adults. A further expansion of the network was announced in 2014. The application period will run until January 2015 and new sites will be selected by June 2015. These sites will officially join the network in 2016. 2015 Update: 37 applications were received, scored and evaluated by a selection committee. At the end of this process, 13 sites with the highest ranking were selected to join the ECFS-CTN. Four new countries are involved (Ireland, Israel, Poland and Switzerland) and the network now covers around 17500 adult and pediatric CF patients in 15 countries and 43 sites. 3. What does the ECFS-CTN do? The aim of the ECFS-CTN is to intensify the clinical research in the field of CF in terms of quality and quantity to ensure people with CF in Europe and beyond have access to effective treatment as quickly as possible. This is done by: • Involving and cooperating with the patient organizations. Since the start of the ECFS-CTN, a close cooperation has been setup with patient organizations (national and European level). A patient organization representative, appointed by CF Europe, is member of the network’s executive committee and participates in 2-weekly